Mitapivat sulfate is a Small Molecule owned by Agios Pharmaceuticals, and is involved in 22 clinical trials, of which 13 were completed, 8 are ongoing, and 1 is planned.

Mitapivat sulfate acts as PKR activator. Mutations in PKR cause defects in red cell glycolysis and leads to a hematological inborn error metabolism known as pyruvate kinase deficiency. Mitapivat is a pyruvate kinase activator that acts by allosterically binding to the pyruvate kinase tetramer and increasing pyruvate kinase (PK) activity. The red blood cell (RBC) form of pyruvate kinase (PK-R) is mutated in PK deficiency, which leads to reduced adenosine triphosphate (ATP), shortened RBC lifespan, and chronic hemolysis.

The revenue for Mitapivat sulfate is expected to reach a total of $6.6bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Mitapivat sulfate NPV Report.

Mitapivat sulfate is originated and owned by Agios Pharmaceuticals.

Mitapivat sulfate Overview

Mitapivat sulfate (Pyrukynd) act as anti anemic agent. It is formulated as tablets, film coated tablets for oral route of administration. Pyrukynd is indicated for the treatment of hemolytic anemia (a disorder in which red blood cells are destroyed faster than they can be made) in adults with pyruvate kinase (PK) deficiency.

Mitapivat sulfate (AG-348) is under development for the treatment of a form of hemolytic anemia known as pyruvate kinase deficiency, alpha thalassemia and beta thalassemia and sickle cell disease. The drug candidate is a quinolone sulfonamide administered through oral route as a capsule and tablet. It targets pyruvate kinase receptor (PKR).

Agios Pharmaceuticals Overview

Agios Pharmaceuticals (Agios) is a biopharmaceutical company. The company focuses on the discovery and development of investigational medicines for the treatment of cancer and rare genetic metabolic disorders. Its pipeline products include AG-946, a novel, next-generation PKR activator used for the treatment of hemolytic anemias and other indications, including SCD and anemia associated with low- to intermediate-risk myelodysplastic syndrome. The company also provides Mitapivat which is an activator of both wild-type and a variety of mutant pyruvate kinase that is used against hemolytic anemias. Agios is headquartered in Cambridge, Massachusetts, the US.

The operating loss of the company was US$371.8 million in FY2021, compared to an operating loss of US$335.9 million in FY2020. The net profit of the company was US$1,604.7 million in FY2021, compared to a net loss of US$327.4 million in FY2020. The company reported revenues of US$3.5 million for the third quarter ended September 2022, a decrease of 37% over the previous quarter.

Quick View – Mitapivat sulfate

Report Segments
  • Innovator (NME)
Drug Name
  • Mitapivat sulfate
Administration Pathway
  • Oral
Therapeutic Areas
  • Hematological Disorders
Key Companies
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.